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Repeated hUCB injections may improve prognosis of children with deadly inherited disorder
Date:3/28/2014

Putnam Valley, NY. (Mar. 28 2014) New insight has been gained into treating an inherited disorder that creates serious neurological and behavioral disabilities in children and usually leads to death in the teen years.

In a recent study into the effects of human umbilical cord blood mononuclear cells (hUCB MNCs) when they are injected to counter the symptoms and progression of Sanfilippo syndrome type III B (MPS III B), researchers found that repeated injections into laboratory mice modeled with the disorder had clear benefits for the mice receiving multiple injections over control groups that received single injections of either a high or low dose of cells.

The study will be published in a future issue of Cell Transplantation but is currently freely available on-line as an unedited early e-pub at: http://www.ingentaconnect.com/content/cog/ct/pre-prints/content-ct1121willing.

MPS III B results from a genetically programmed deficit of the Naglu enzyme. The deficit creates a build-up of heparan sulfate - a complex carbohydrate - that accumulates in lysosomes, cells that are responsible for waste disposal. With MPS III B, accumulations of heparin sulfate in the tissues are not eliminated and the accumulation causes damage to multiple organs, including the brain.

"Cell therapy has recently received attention as a potential treatment for lysosomal storage diseases," said study lead author Dr. Allison E. Willing, of the Center of Excellence for Aging and Brain Repair in the Morsani College of Medicine at the University of South Florida. "We have previously shown that a single hUCB injection into the cerebral ventricle of pre-symptomatic mice, or intravenous cell delivery at different disease stages, had a beneficial effect on the enzyme deficient mice. In the current study, we examined whether administering repeated doses of hUCB MNCs
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Contact: Robert Miranda
cogcomm@aol.com
Cell Transplantation Center of Excellence for Aging and Brain Repair
Source:Eurekalert

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